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10 Gene Therapy Companies Unignorable in 2019

Applied Genetic Technologies
Applied Genetic Technologies

Applied Genetic Technologies Corp. (AGTC) is a clinical-stage biotechnology company developing gene therapies, with an initial focus in ophthalmology.

The Company’s investigational gene therapies are:

-- Gene therapy for Achromatopsia, under two parallel phase I/II studies, namely CNGB3 trial and CNGA3 trial. In the CNGB3 trial, a total of 10 ten patients were enrolled as of last November. The company expects to complete the dose escalation portion of the CNGB3 trial in the first quarter of 2019.

-- Gene therapy for X-linked Retinitis Pigmentosa (XLRP), under phase I/II study. Eight patients have been enrolled in this study, and the dose escalation portion of the study is expected to be completed in the first quarter of 2019.

-- In December 2018, the Company reported topline interim six-month data from its phase I/II trial of its gene therapy for X-linked retinoschisis (XLRS). Results from the study showed that the gene therapy was safe and well-tolerated, but no signs of clinical activity were observed at six-months.

Data readouts from the trials can be expected this year.

AGTC closed Wednesday’s (Jan.9, 2019) trading at $3.05, up 3.04%.

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